PB2308: QUALITY OF LIFE AND CLINICAL OUTCOMES IN ADULT PATIENTS WITH CHRONIC IMMUNE THROMBOCYTOPENIA (CITP) DURING TREATMENT WITH ROMIPLOSTIM IN A REAL-WORLD SETTING
Authors: T. Ionova, O. Vinogradova, E. Markova, L. Mukha, N. Novitskaya, M. Pankrashkina, T. Shelekhova, D. Sherstnev, A. Proydakov, E. Lyurova, T. Pospelova, T. Babaeva, N. Bulieva, G. Kuchma, E. Andreevskaya, M. Frolova, E. Zinina, K. Trizna, I. Shestopalova, T. Shneyder, L. Khvorostenko, A. Kuchin, I. Mulina, S. Volkova, S. Zakharov, N. Porfirieva, T. Nikitina, S. Gritsaev
Journal: HemaSphere (2022)

Background: Chronic primary immune thrombocytopenia (cITP) is a rare autoimmune disease which needs lifelong therapy to prevent bleeding and is accompanied with impaired quality of life (QoL) due to hemorrhagic syndrome, psychological concerns and limitations in patients’ daily life. Fatigue with its negative impact on QoL is of tremendous concern for patients with cITP. Aims: We aimed to explore QoL and fatigue changes as well as to examine platelet response in patients with cITP during treatment with romiplostim in a real-world setting. Methods: Adult patients with cITP who have been assigned romiplostim treatment were enrolled in multicenter observational prospective study. Romiplostim was administered in accordance with prescription. Patients filled out RAND SF-36 and FACT-Th6 questionnaires for QoL assessment and FACIT-Fatigue tool for fatigue assessment at baseline and at 3, 6 and 12 months after romiplostim treatment start. Fatigue level was categorized according to the quartiles of the FACIT-Fatigue scores as follows: low (Q4, 40-52), low-medium (Q3, 27-39), medium-high (Q2, 14-26), or high (Q1, 0-13). For statistical analysis of patient-reported outcomes (PRO) Mann-Whitney test, GEE and MacNemar test were applied. Adjustment to age, gender, severity of cITP and baseline QoL/fatigue for GEE was made. Kaplan-Meyer method was used to measure time till the first platelet response from romiplostim treatment start. Results: In total, 60 patients with cITP from 17 centers in Russia were enrolled in the study: mean age – 51.9±15.4 (range, 25-85); 70% were females; 71.7% patients had comorbidities. Four patients were splenectomized (6.7%). Median of disease duration was 25.1 months (range, 12.6-765.1). Majority of patients experienced hemorrhagic syndrome (96.7%). Median platelet count before romiplostim was 20 х109/l. Median number of treatments before romiplostim was 1 (range, 0-6). At baseline cITP patients experienced meaningful QoL impairment by all SF-36 scales excluding pain as compared to healthy controls (p<0.05). Integral QoL index was 0.285 for patients vs 0.432 for healthy controls (p<0.001); 39% patients had critical/severe QoL impairment before romiplostim onset. Mean FACT-Th6 score was 11.2±5.5 (range, 0-22). Мean FACIT-Fatigue score was 33.0±10.2 (range, 7–49); 22.4% of patients had high/medium-high level of fatigue. At median follow-up of 9.7 months (IQR, 7.2–12), 98.3% patients achieved overall platelet response; median time to first platelet response was 4 weeks (95%CI: 3–5). Median platelet count at 3 months of treatment was 104 х109/l. During 12 months of romiplostim treatment dramatic meaningful QoL improvement by all SF-36 scales (GEE, p<0.001) and by FACT-Th6 score (GEE, 11.4 at baseline vs 17.1 at 12 months, p<0.001) was demonstrated. Fatigue severity reduced during romiplostim treatment: FACIT-Fatigue score increased from 33.1 at baseline to 42.7 at 12 months (GEE, p<0.001); the number of patients with high/medium-high level of fatigue significantly decreased from 22.4% at baseline to 6.8% after 3 months of treatment (p=0.01). Summary/Conclusion: The results obtained demonstrated high response rates to romiplostim treatment in cITP patients and significant positive changes in QoL and fatigue level during therapy in a real-world evidence study. Benefits of romiplostim treatment was confirmed both from physician’s and patient’s perspective.